Advanced Malignant Solid NeoplasmPhase 1NCT01303341
Riluzole and Sorafenib Tosylate in Treating Patients With Advanced Solid Tumors or Melanoma
A Phase 1 study testing Laboratory Biomarker Analysis, Pharmacological Study and Riluzole in people with Advanced Malignant Solid Neoplasm and Recurrent Melanoma, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
This phase I trial studies the side effects and best dose of sorafenib tosylate when given together with riluzole in treating patients with solid tumors or melanoma that has spread to other places in the body and usually cannot be cured or controlled with treatment. Riluzole may stop or slow the growth of tumor cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Malignant Solid Neoplasm, Recurrent Melanoma, Refractory Malignant Solid Neoplasm
- What is being tested?
- Laboratory Biomarker Analysis, Pharmacological Study and Riluzole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Feb 2011to 11 Sep 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have histologically proven solid tumors (Phase I) with biopsiable tumor (expansion cohort) refractory to standard therapy or for whom no standard therapy exists or who decline standard therapy
- Eastern Cooperative Oncology Group (ECOG) performance status =< 2
- Patients must be willing and able to sign informed consent
- Unlimited prior therapies are permitted for patients enrolled in the dose escalation phase of the study; patients in the expansion cohort of the study may not have any prior therapy with riluzole or sorafenib and must have biopsiable tumor
- Patients may have measurable or evaluable disease
- Absolute neutrophil count (ANC) >= 1,500/uL
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- Platelets >= 100,000/uL
- Total bilirubin =< 1.5 x institutional upper limit of normal (ULN)
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x institutional ULN
- International normalized ratio (INR) =< 1.5 x institutional ULN
- Creatinine =< 2 x ULN
- Patients with brain lesions that have been treated with whole brain radiotherapy and are clinically stable for at least 4 weeks, are not taking steroids and are not receiving enzyme-inducing anticonvulsants will be eligible
Who cannot join
- Serious concomitant systemic disorders (including active infections) that would compromise the safety of the patient or compromise the patient's ability to complete the study, at the discretion of the investigator
- For patients who have received gamma knife or stereotactic radiosurgery, a 2 week washout is required; patients who have had other types of radiotherapy, chemotherapy or biologic agents within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 3 weeks earlier to =< grade 1; at least 4 weeks must have elapsed since any major surgery; patients with prostate cancer may continue to receive hormonal therapy
- History of allergic reactions attributed to riluzole or sorafenib
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal, barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 2 weeks after discontinuation of riluzole and/or sorafenib; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately; pregnant (positive pregnancy test) or lactating patients cannot participate
- Known human immunodeficiency virus (HIV) infection or known history of active hepatitis B or C infection
- Current, recent (within 4 weeks of the first treatment of this study), or planned participation in an experimental drug study (prevention trials are permitted if the trial is not testing a novel experimental agent)
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- Cardiac disease: congestive heart failure > class II New York Heart Association (NYHA); patients must not have unstable angina (anginal symptoms at rest) or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
- History of stroke within six months
- Clinically significant peripheral vascular disease
- Cardiac ventricular arrhythmias requiring anti-arrhythmic therapy
- Uncontrolled hypertension, defined as systolic blood pressure > 150 mm Hg or diastolic blood pressure > 90 mm Hg, despite optimal medical management
- Active clinically serious infection > Common Terminology Criteria for Adverse Events (CTCAE) grade 2
- Any of the following within 6 months prior to first dose of treatment: myocardial infarction, symptomatic coronary artery disease (severe or unstable angina), artery bypass graft, uncontrolled arrhythmias, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, or pulmonary embolus
- Pulmonary hemorrhage/bleeding event >= CTCAE grade 2 within 4 weeks of first-dose of study drug
- Any other hemorrhage/bleeding event >= CTCAE grade 3 within 4 weeks of first dose of study drug
- Evidence or history of bleeding diathesis or coagulopathy
- Major surgery or significant traumatic injury within 4 weeks of first study drug
- The eligibility of patients taking medications that are potent modulators of cytochrome P450, family 3, subfamily A, polypeptide 4 (CYP3A4), cytochrome P450, family 2, subfamily B, polypeptide 6 (CYP2B6), subfamily 2, polypeptide 8 (2C8) will be determined following a review of their case by the principal investigator; every effort should be made to switch patients taking such agents or substances to other medications
- Any condition that impairs the patient's ability to swallow whole pills
- Any malabsorption problem
- Anticipation of need for major surgical procedure during the course of the study
- History of abdominal fistula, gastrointestinal perforation, or intra-abdominal abscess within 6 months prior to day 1 of treatment
- Serious, non-healing wound, ulcer, or bone fracture
- Inability to comply with study and/or follow-up procedures
- Anticoagulation with Lovenox (enoxaparin) is permitted, however, patients on anticoagulation with warfarin are not permitted on this study
Trial locations and contacts
1 site in 1 country.
United States1 site
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903
About this study
Official title: A Phase I Trial of Riluzole and Sorafenib in Patients With Advanced Solid Tumors and Melanoma
This phase I trial studies the side effects and best dose of sorafenib tosylate when given together with riluzole in treating patients with solid tumors or melanoma that has spread to other places in the body and usually cannot be cured or controlled with treatment. Riluzole may stop or slow the growth of tumor cells. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving riluzole together with sorafenib tosylate may kill more tumor cells.
Read the detailed description
PRIMARY OBJECTIVES:
- To define a safe dose of sorafenib (sorafenib tosylate) to combine with riluzole in the treatment of patients with all types of solid tumors refractory to standard therapy or for whom no standard therapy exists.
SECONDARY OBJECTIVES:
- To examine the correlation of clinical or radiologic response with signaling through the mitogen-activated protein kinase (MAPK) and phosphoinositide 3-kinase (PI3K)/protein kinase B (AKT) pathways.
II. To determine if response to therapy with riluzole and sorafenib correlates with expression levels of B-cell lymphoma (BCL)-2, myeloid cell leukemia (MCL)-1, or BCL2-like 11 (apoptosis facilitator) (BIM).
III. To characterize the pharmacokinetics of the combination of riluzole with sorafenib and determine if any drug-drug interactions exist.
IV. To evaluate the microvesicle (an inter-cellular communication approach which may cargo proteins, ribonucleic acids [RNAs] and deoxyribonucleic acids [DNAs] to its host cell) quantification difference between pre-treatment and post-treatment peripheral blood samples of patients.
OUTLINE: This is a dose-escalation study of sorafenib tosylate.
Patients receive riluzole orally (PO) twice daily (BID) and sorafenib tosylate PO once daily (QD) or BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study therapy, patients are followed up for approximately 2-3 years.
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Melanoma, Refractory Malignant Solid Neoplasm, Stage III Cutaneous Melanoma AJCC v7, Stage IIIA Cutaneous Melanoma AJCC v7, Stage IIIB Cutaneous Melanoma AJCC v7
- Being tested
- Laboratory Biomarker Analysis, Pharmacological Study, Riluzole, Sorafenib Tosylate
- Medical subject headings
- Melanoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laboratory Biomarker AnalysisOther
Correlative studies
Correlative studies
RiluzoleDrug
Also called Rilutek
Given PO
Sorafenib TosylateDrug
Also called BAY 43-9006 Tosylate, BAY 54 9085, BAY 54-9085, BAY 549085
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (riluzole and sorafenib tosylate) | Experimental | Patients receive riluzole PO BID and sorafenib tosylate PO QD or BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Receives: Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; Drug: Riluzole; Drug: Sorafenib Tosylate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum-tolerated dose of sorafenib tosylate and riluzole in patients with all types of solid tumors
Maximum tolerated dose is defined as the first dose level at which exactly 2/6 patients experience dose limiting toxicity, or at which 1/6 experience dose limiting toxicity and (due to de-escalation) at least 2/3 or 3/6 patients treated with the next higher dose level had dose limiting toxicity.
Measured: 28 days
Secondary outcomes (6)
Suppression of MAPK and PI3K/AKT pathways
Will examine the correlation of clinical or radiologic response with signaling.
Measured: Up to 3 years
Change in BCL-2 expression
Appropriate parametric (such as paired t-test) or nonparametric (such as Wilcoxon signed rank test) methods will be used.
Measured: Baseline to 3 years
Change in MCL-1 expression
Appropriate parametric (such as paired t-test) or nonparametric (such as Wilcoxon signed rank test) methods will be used.
Measured: Baseline to 3 years
Change in BIM expression
Appropriate parametric (such as paired t-test) or nonparametric (such as Wilcoxon signed rank test) methods will be used.
Measured: Baseline to 3 years
Pharmacokinetic parameters of the combination of riluzole with sorafenib tosylate
Will be assessed from blood samples.
Measured: On days 2, 8, 10, and 15 of each course
Change in microvesicle quantification
Will be assessed from blood samples.
Measured: Baseline to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2011 (estimated)
- Study start18 Feb 2011
- Primary completion1 May 2012
- Study completion11 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Advanced Malignant Solid NeoplasmStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Janice M Mehnert, Principal investigator, Rutgers Cancer Institute of New Jersey
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01303341
- Sponsor's protocol ID
- NCI-2011-02597
- Other IDs
- NCI-2011-02597, CDR0000695355, 090906, 8850, 8850, P30CA072720, R01CA149627, U01CA132194
- Status last verified
- Sep 2026
- First submitted
- 22 Feb 2011
- Last updated
- 15 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
Spencer KR, Portal DE, Aisner J, Stein MN, Malhotra J, Shih W, Chan N, Silk AW, Ganesan S, Goodin S, Gounder M, Lin H, Li J, Cerchio R, Marinaro C, Chen S, Mehnert JM. A phase I trial of riluzole and sorafenib in patients with advanced solid tumors: CTEP #8850. Oncotarget. 2023 Apr 10;14:302-315. doi: 10.18632/oncotarget.28403.
PubMed 37036756
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial of Riluzole and Sorafenib in Patients With Advanced Solid Tumors and Melanoma
- ClinicalTrials.gov ID
- NCT01303341
- Acronym / sponsor ID
- NCI-2011-02597
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Melanoma, Refractory Malignant Solid Neoplasm, Stage III Cutaneous Melanoma AJCC v7, Stage IIIA Cutaneous Melanoma AJCC v7, Stage IIIB Cutaneous Melanoma AJCC v7
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 35 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 18 Feb 2011 (actual)
- Primary completion
- 1 May 2012 (actual)
- Study completion
- 11 Sep 2027 (estimated)
- First posted
- 24 Feb 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase I trial studies the side effects and best dose of sorafenib tosylate when given together with riluzole in treating patients with solid tumors or melanoma that has spread to other places in the body and usually cannot be cured or controlled with treatment. Riluzole may stop or slow the growth of tumor cells. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving riluzole together with sorafenib tosylate may kill more tumor cells.
Detailed description
PRIMARY OBJECTIVES:
- To define a safe dose of sorafenib (sorafenib tosylate) to combine with riluzole in the treatment of patients with all types of solid tumors refractory to standard therapy or for whom no standard therapy exists.
SECONDARY OBJECTIVES:
- To examine the correlation of clinical or radiologic response with signaling through the mitogen-activated protein kinase (MAPK) and phosphoinositide 3-kinase (PI3K)/protein kinase B (AKT) pathways.
II. To determine if response to therapy with riluzole and sorafenib correlates with expression levels of B-cell lymphoma (BCL)-2, myeloid cell leukemia (MCL)-1, or BCL2-like 11 (apoptosis facilitator) (BIM).
III. To characterize the pharmacokinetics of the combination of riluzole with sorafenib and determine if any drug-drug interactions exist.
IV. To evaluate the microvesicle (an inter-cellular communication approach which may cargo proteins, ribonucleic acids [RNAs] and deoxyribonucleic acids [DNAs] to its host cell) quantification difference between pre-treatment and post-treatment peripheral blood samples of patients.
OUTLINE: This is a dose-escalation study of sorafenib tosylate.
Patients receive riluzole orally (PO) twice daily (BID) and sorafenib tosylate PO once daily (QD) or BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study therapy, patients are followed up for approximately 2-3 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (riluzole and sorafenib tosylate) | Experimental | Other: Laboratory Biomarker Analysis, Other: Pharmacological Study, Drug: Riluzole, Drug: Sorafenib TosylatePatients receive riluzole PO BID and sorafenib tosylate PO QD or BID on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Patients must have histologically proven solid tumors (Phase I) with biopsiable tumor (expansion cohort) refractory to standard therapy or for whom no standard therapy exists or who decline standard therapy
- Eastern Cooperative Oncology Group (ECOG) performance status =< 2
- Patients must be willing and able to sign informed consent
- Unlimited prior therapies are permitted for patients enrolled in the dose escalation phase of the study; patients in the expansion cohort of the study may not have any prior therapy with riluzole or sorafenib and must have biopsiable tumor
- Patients may have measurable or evaluable disease
Key exclusion criteria (25 listed)
- Serious concomitant systemic disorders (including active infections) that would compromise the safety of the patient or compromise the patient's ability to complete the study, at the discretion of the investigator
- For patients who have received gamma knife or stereotactic radiosurgery, a 2 week washout is required; patients who have had other types of radiotherapy, chemotherapy or biologic agents within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 3 weeks earlier to =< grade 1; at least 4 weeks must have elapsed since any major surgery; patients with prostate cancer may continue to receive hormonal therapy
- History of allergic reactions attributed to riluzole or sorafenib
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal, barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 2 weeks after discontinuation of riluzole and/or sorafenib; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately; pregnant (positive pregnancy test) or lactating patients cannot participate
- Known human immunodeficiency virus (HIV) infection or known history of active hepatitis B or C infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (riluzole and sorafenib tosylate) |
| Pharmacological StudyCorrelative studies | Other | Treatment (riluzole and sorafenib tosylate) |
| RiluzoleAlso: RilutekGiven PO | Drug | Treatment (riluzole and sorafenib tosylate) |
| Sorafenib TosylateAlso: BAY 43-9006 Tosylate, BAY 54 9085, BAY 54-9085, BAY 549085Given PO | Drug | Treatment (riluzole and sorafenib tosylate) |
Primary outcome
Maximum-tolerated dose of sorafenib tosylate and riluzole in patients with all types of solid tumors
Time frame: 28 days
Maximum tolerated dose is defined as the first dose level at which exactly 2/6 patients experience dose limiting toxicity, or at which 1/6 experience dose limiting toxicity and (due to de-escalation) at least 2/3 or 3/6 patients treated with the next higher dose level had dose limiting toxicity.
6 secondary outcomes
Suppression of MAPK and PI3K/AKT pathways
Time frame: Up to 3 years
Change in BCL-2 expression
Time frame: Baseline to 3 years
Change in MCL-1 expression
Time frame: Baseline to 3 years
Change in BIM expression
Time frame: Baseline to 3 years
Pharmacokinetic parameters of the combination of riluzole with sorafenib tosylate
Time frame: On days 2, 8, 10, and 15 of each course
Change in microvesicle quantification
Time frame: Baseline to 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2026.
- Derived from this study: PubMed 37036756Spencer KR, Portal DE, Aisner J, Stein MN, Malhotra J, Shih W, Chan N, Silk AW, Ganesan S, Goodin S, Gounder M, Lin H, Li J, Cerchio R, Marinaro C, Chen S, Mehnert JM. A phase I trial of riluzole and sorafenib in patients with advanced solid tumors: CTEP #8850. Oncotarget. 2023 Apr 10;14:302-315. doi: 10.18632/oncotarget.28403.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Janice M Mehnert principal investigator
Rutgers Cancer Institute of New Jersey
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Laboratory Biomarker Analysis, Pharmacological Study and Riluzole in people with Advanced Malignant Solid Neoplasm and Recurrent Melanoma, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum-tolerated dose of sorafenib tosylate and riluzole in patients with all types of solid tumors (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01303341, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2026.